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| Sponsors and Collaborators: |
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) University of Florida |
|---|---|
| Information provided by: | Office of Rare Diseases (ORD) |
| ClinicalTrials.gov Identifier: | NCT00004533 |
Purpose
OBJECTIVES:
I. Determine the maximum tolerated dose of recombinant adeno-associated virus-CFTR vector in patients with cystic fibrosis.
II. Assess the safety of this gene therapy in these patients.
III. Assess the in vivo gene transfer of this vector in these patients.
IV. Assess the CFTR gene expression and physiologic activity following gene transfer in these patients.
V. Assess the clinical impact of CFTR gene expression following gene transfer in these patients.
VI. Monitor patient immune response directed against CFTR or vector components following vector administration.
| Condition | Intervention | Phase |
|---|---|---|
|
Cystic Fibrosis |
Drug: Adeno-associated virus-CFTR vector |
Phase I |
| Study Type: | Interventional |
| Study Design: | Treatment, Randomized, Double-Blind, Placebo Control |
| Estimated Enrollment: | 19 |
| Study Start Date: | August 1999 |
PROTOCOL OUTLINE:
This is a randomized, dose escalation, double blind, placebo controlled, multicenter study. Patients are randomized to receive either adeno-associated virus-CFTR (AAV-CFTR) vector or placebo.
Patients undergo tests on days -10 to -4 to rule out adenovirus and adeno-associated virus infections. Patients then receive AAV-CFTR vector intranasally to the right or left inferior turbinates and placebo to the other side of the nose. The next day, patients receive an endobronchial dose of AAV-CFTR vector to the superior segment of the right lower lobe.
Cohorts of 2-4 patients each receive escalating doses of AAV-CFTR vector until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose level immediately preceding the dose at which at least 2 subjects develop dose limiting toxicity.
Patients are followed at day 10, then at 1, 2, 3, 6, 9, and 12 months.
Eligibility| Ages Eligible for Study: | 15 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Diagnosis of cystic fibrosis (CF) with mild to moderate lung disease; FVC at least 60% predicted; Schwachman-Kulczycki score at least 65; Sweat chloride greater than 60 mEq/L
All CF genotypes allowed
No sputum colonization with Burkholderia cepacia or another multiply resistant organism
No recurrent or intermittent hemoptysis; At least 1 year since significant hemoptysis requiring transfusion or embolization OR Coughing of greater than 30 mL of blood more often than once a week
--Prior/Concurrent Therapy--
At least 30 days since hospitalization or home intravenous antibiotic therapy for pulmonary exacerbation
No concurrent investigational drugs or therapy
--Patient Characteristics--
Other: No concurrent cigarette smoking; Not pregnant or nursing; Negative pregnancy test; Fertile patients must use effective contraception
Contacts and Locations More Information
| Study ID Numbers: | 199/14137, UF-G-037-96, TGC-AAV-9502, UF-G-37-1996 |
| Study First Received: | January 28, 2000 |
| Last Updated: | June 23, 2005 |
| ClinicalTrials.gov Identifier: | NCT00004533 History of Changes |
| Health Authority: | United States: Federal Government |
|
cardiovascular and respiratory diseases cystic fibrosis genetic diseases and dysmorphic syndromes rare disease |
|
Virus Diseases Digestive System Diseases Genetic Diseases, Inborn Respiratory Tract Diseases Cystic Fibrosis Fibrosis |
Lung Diseases Respiration Disorders Rare Diseases Infant, Newborn, Diseases Pancreatic Diseases |
|
Pathologic Processes Digestive System Diseases Genetic Diseases, Inborn Respiratory Tract Diseases Cystic Fibrosis |
Fibrosis Lung Diseases Infant, Newborn, Diseases Pancreatic Diseases |
